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MedNess: bite-size biopharma and medtech news

17th February, 2021
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MedNess This Week
HIGHLIGHTS
Onco-I-Analyse
Libtayo becomes the first immunotherapy drug approved for basal cell carcinoma
On 9th February, the US FDA granted regular and accelerated approval to Regeneron’s cemiplimab-rwlc (Libtayo), a PD-1 inhibitor, for the treatment of patients with locally advanced basal cell carcinoma (laBCC) and metastatic BCC (mBCC), respectively, who had progressed on prior hedgehog pathway inhibitor (HHI) or considered inappropriate/intolerant for HHI.
Background: Basal cell carcinoma is the most common skin cancer in the US with an annual incidence of nearly 2 million Americans, exceeding the combined incidence of all other cancer types. Although associated with good prognosis, BCC is the second most common type of skin cancer. It is rarely metastatic but can cause considerable local destruction along with disfigurement.
For recurrent and metastatic BCC, HHI are the only treatment options. However, patients with advanced BCC usually develop resistance which limits the duration of response.
Details: The approval was based on the non-randomized Phase 2 study evaluating Libtayo in patients with mBCC or unresectable laBCC either progressing on HHI or were intolerant to prior HHI therapy.
Confirmed ORR of 29% (5CR, 19PR) was demonstrated amongst 84 patients with laBCC, 79% of whom maintained the response for ≥6 months. With longer follow-up, ORR in laBCC increased to 31%. Among 28 patients with mBCC, confirmed ORR of 21% (All PR) was observed with all patients maintaining the responses for ≥6 months. Median duration of response and median overall survival were not reached in both cases.
Serious adverse reactions occurred in 32% patients; 13% patients discontinued the study due to AEs.
The FDA assessment used the Assessment Aid and the application was granted priority review.
Implications: Although Libtayo is the 6th checkpoint inhibitor (CHKPTi) to enter the US market, dominated by Merck’s Keytruda, it could possibly carve out a niche market for itself as the first immunotherapy drug approved for BCC. In 2018, Libtayo was also the first CHKPTi to be approved for locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC).
Approvals in CSCC and BCC reinforces Regeneron’s position in dermato-oncology with few analysts predicting sales of the drug to eventually hit $2 billion.
 
Collated by : Shilpa Rawal, PhD
Drug Approvals
European Commission Approves TUKYSA® (tucatinib) for the Treatment of Patients with Locally Advanced or Metastatic HER2-Positive Breast Cancer

“This approval is a significant advancement for patients in Europe, who will for the first time have an approved medicine demonstrating a survival benefit for HER2-positive metastatic breast cancer after disease progression following two standard anti-HER2 treatment regimens,” said Prof. Dr. Med Volkmar Mueller, Deputy Director at the University Medical Center Hamburg-Eppendorf, Hamburg, Germany and investigator for the pivotal trial. “In the HER2CLIMB pivotal trial, the tucatinib combination regimen improved overall and progression-free survival compared to trastuzumab and capecitabine alone, including in patients with active, untreated or progressing brain metastases, a population with significant unmet need.”
Regulatory News
Tisotumab Vedotin BLA submitted to the U.S. FDA for Patients with Recurrent or Metastatic Cervical Cancer
“In the pivotal phase 2 study, tisotumab vedotin induced clinically meaningful and durable responses in this difficult to treat cervical cancer patient population, with a manageable and tolerable safety profile. Today’s submission marks an important milestone for tisotumab vedotin and a potential advance for patients with recurrent or metastatic cervical cancer for whom there is a high unmet need for effective new therapies,” said Roger Dansey, M.D., Chief Medical Officer at Seagen. “We look forward to working with the FDA on the review of the application.”
FDA Breakthrough Therapy designations for investigational STAMP inhibitor asciminib (ABL001) in chronic myeloid leukemia
  • Asciminib targets the ABL myristoyl pocket (STAMP)
  • Granted Breakthrough Therapy designation (BTD) by the FDA for the treatment of adult Ph+ CML patients in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs).
  • Asciminib was also granted BTD for the treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.
  • FDA designations for asciminib was based on the pivotal, Ph 3 ASCEMBL trial of asciminib vs Bosulif® (bosutinib)* in patients with Ph+ CML in CP previously treated with two or more TKIs and a Ph 1 trial that included patients with Ph+ CML, some of them harboring the T315I mutation.
Click here for more Regulatory News
Trial Results
KEYTRUDA® + LENVIMA® Demonstrated Superior PFS and OS Versus Sunitinib as First-Line Treatment for Patients with Advanced RCC
“Continued efforts to improve outcomes in patients with advanced renal cell carcinoma (RCC) are critical, considering that the number of people diagnosed with the disease has more than doubled over the last 50 years, and almost one-third of these patients are diagnosed at an advanced stage,” said Dr. Robert Motzer, Medical Oncologist, Kidney Cancer Section Head, Genitourinary Oncology Service, Memorial Sloan Kettering Cancer Center. “KEYTRUDA plus LENVIMA demonstrated a median progression-free survival of nearly two years, and seven in 10 patients experienced an objective response. This combination also significantly improved overall survival compared with sunitinib, with a 34% reduction in risk of death. These results suggest that this combination has the potential to impact clinical practice for this type of devastating cancer.”
FAILED TRIAL: Ph 3 CanStem303C Study of Napabucasin Fails to Reach Primary Endpoints of OS improvement in Patients with Previously Treated mCRC
"Patients with metastatic colorectal cancer have a high unmet medical need, and our hope was to develop a new treatment option for this population. We are disappointed with the results of this Phase 3 trial and would like to express gratitude to the trial participants, investigators and staff for their efforts and contributions to the study," said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Dainippon Pharma Oncology (SDP Oncology). "SDP Oncology is committed to continuing our pursuit of advancing our pipeline to bring forward innovative treatments for patients with cancer."
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Trial/Program Status
TRYbeCA-1 Ph 3 Trial in 2L Pancreatic Cancer to Continue to Final Analysis as per IDMC recommendations
“This IDMC review was the first combined efficacy and safety review. We are pleased that no safety issues were raised.” said Gil Beyen, Chief Executive Officer of ERYTECH Pharma. “The trial will now continue to the final efficacy analysis. With 512 patients enrolled, the trial has approximately 90% power to detect the trial’s design hazard ratio of 0.725. Second-line pancreatic cancer is a devastating disease and remains a large unmet medical need. We are hopeful that TRYbeCA-1 can confirm the survival benefit we observed in the Phase 2b clinical trial and eryaspase can be a potential treatment for these patients. We look forward to sharing final results later this year.”
Click here for more Trial Statuses
Collated by : Richa Tewari, PhD
Genes and Therapy
The first investigational cell therapy for Type 1 Diabetes receives clearance for clinical trials
Vertex Pharmaceuticals receives FDA clearance for clinical trials for its investigational new drug (IND) VX-880 against Type 1 Diabetes (T1D).  VX-880 is an allogeneic human stem cell-derived, fully differentiated pancreatic islet cell therapy that will be evaluated in patients with impaired hypoglycemic awareness and severe hypoglycemia.  The Phase 1/2 clinical trial is set to begin in the first half of this year.  The single-arm, open-label study will recruit 17 patients who will be infused with different doses of fully differentiated, functional islet cells.  They will also be administered long-term immunosuppressive therapy to prevent immune rejection of pancreatic islet cells.  T1D is an autoimmune condition where insulin-producing pancreatic islet cells are destroyed resulting in impaired blood glucose control. Presently, insulin management is the only prophylaxis available for this disease.
First gene therapy, OAV201, against Rett Syndrome to seek FDA approval for clinical trials
Novartis Gene Therapies plans to submit its IND application to FDA by the end of this year, for OAV201, a gene therapy candidate for Rett Syndrome.  Upon approval, the company hopes to begin clinical trials by late this year or early next year. 
Rett Syndrome is a rare neurodevelopmental disorder that causes progressive loss of speech and motor skills.  It is usually caused by mutations in an X-linked gene, MECP2, and therefore primarily affects girls.  Presently there is no cure for Rett Syndrome.  OAV201 uses an engineered viral vector to incorporate the non-mutated version of the MECP2 gene into patient cells to produce a healthy protein.  This therapy was originally developed by AveXis, which has been acquired by Novartis and renamed Novartis Gene Therapies.  An initial IND application for OAV201 was withdrawn in 2019 by AveXis due to data inaccuracies.  Now with strong preclinical data, the company expects IND clearance.
Click here for more on Genes and Therapy
Medness Business
Onco-News
AbbVie and Caribou Biosciences Announce Collaboration and License Agreement for CAR-T Cell Products
“We are excited to partner with AbbVie on the development of new CAR-T cell therapies. This collaboration validates Caribou’s differentiated next-generation CRISPR genome editing technologies that provide best-in-class efficiency and specificity,” said Rachel Haurwitz, Ph.D., President and Chief Executive Officer of Caribou. “We believe AbbVie is an ideal partner for Caribou as we expand upon the number of targets and diseases addressable by our technologies. Genome-edited CAR-T cell therapies hold tremendous potential for patients, and this partnership accelerates our ability to address significant unmet medical need.”
4D pharma, Merck and Pfizer to evaluate MRx0518 in combination with BAVENCIO® for the treatment of locally advanced or metastatic urothelial carcinoma
“With this second clinical trial collaboration for MRx0518 with a leading immune checkpoint inhibitor, 4D is able to evaluate MRx0518 in a new combination and earlier treatment setting. Following the promising data already generated in combination with checkpoint inhibitor pembrolizumab in refractory patients, and MRx0518 monotherapy data demonstrating single agent immuno-modulation presented last year at SITC, this collaboration allows us to continue to build a broad understanding of the safety and efficacy of MRx0518 across a range of solid tumors and stages of disease,” said Duncan Peyton, Chief Executive Officer, 4D pharma. “The combination of MRx0518 with BAVENCIO has the potential to further enhance the positive clinical outcomes achieved by BAVENCIO for the significant number of patients in this treatment setting.”
BioPharma and MedTech

BigHat Biosciences raises $19 million in series A funding for automating wet-lab with AI/deep learning to generate high quality next-gen antibodies
BigHat Biosciences (BigHat, San Carlos, CA) announced raising $19 million in series A funding, on 10th February 2021. The investors include lead investor Andreessen Horowitz, along with prior investors 8VC, AME Cloud Ventures, and Innovation Endeavors. The funding was received for its integrated wet-lab and AI/deep learning platform that can manufacture high quality antibodies to scale within days as opposed to months or years by more conventional laboratory approaches.
BigHat’s proprietary platform combines wet-lab cells, which use synthetic biology to produce antibodies discovered in silco, with quality monitoring by AI/deep learning-based program that shortlists antibodies with high structural and functional parameters for the next cycle of in silco fine tuning and antibody production. Reiteration of this AI-guided procedure leads to antibodies with desirable properties of affinity, avidity, solubility etc. to be manufactured to scale against any known/hypothetical antigen. This accelerates candidate discovery and validation, reducing time to enter clinical trials. BigHat established proof of concept by producing a highly neutralizing antibody to SARS-CoV-2 using its platform. For this, it has received the ‘Amgen’s Golden Ticket to MBC BioLabs’ that grants it free access to laboratory space and to Amgen’s experts in science and business.
The newly acquired capital will be used to expand the in-house team to recruit experts in antibody, protein and AI/ML engineering, as well as, computational biologists. The money will also be used to advance their internal pipeline towards clinical trials.

Sandoz acquires GSK’s cephalosporin antibiotics in a $500 million deal
As announced on 11th February 2021, Sandoz (Novartis Div; HQ: Basel, Switzerland) signed an agreement worth $500 million with GSK (HQ Brentford, UK), to acquire GSK’s cephalosporin antibiotics business, which includes 3 key brands- Zinnat®, Zinacef® and Fortum®. This move strengthens Sandoz’s commitment to the antibiotics area of pharmaceuticals.
According to the terms, Sandoz will pay GSK $350 million upon closing of the deal, which is expected to take place by second half of 2021. There will be further $150 million in (undisclosed) milestone payments. The deal is contingent on mutually agreed conditions, including regulatory approvals. Sandoz will acquire global rights to the antibiotics, including Zinnat®, Zinacef® and Fortum®, in 100 worldwide markets, barring for those brands that were previously off-loaded by GSK in US, Australia and Germany. GSK will retain rights in India, Pakistan, Egypt, Japan and China.
GSK will also be supplying Zinnat® to Sandoz under a manufacturing and supply agreement (MSA), till 4 years after deal closure. Sandoz is planning to step up the manufacturing of Zinnat® at its production facilities in Kundl, Austria.
Zinnat® is an orally bioavailable prodrug to the cephalosporin antibiotic, cefuroxime (a beta lactamase resistant broad-spectrum antibiotic). The combined annual sales (2020) of Zinnat®, Zinacef® and Fortum® was ~ $140 million.
“Cephalosporins are the largest antibiotic segment by global sales and acquiring this leading business, including the established global Zinnat® brand, will complement our #1 position in generic penicillins, the other key segment. It will also set us up for additional synergies driven by an increased promotional footprint that will support growth of both the acquired brands and the current existing Sandoz portfolio.”- Sandoz CEO Richard Saynor.
Click here for more on mergers, acquisition and business news
Editors' Desk
Richa Tewari, PhD
Oncology News
Esha Sehanobish, PhD
MedNess Plus
Arundithi Ananthanarayanan
MedNess Reviews
Divyaanka Iyer
BioPharma News
Shilpa Rawal, PhD
Onco I-Analyse
Debarati Banik
HealthIT
Rinki Saha
BioPharma News
Managing Editor
Shalini Roy Choudhury
Genes and Therapy
Managing Editor
Nisha Peter, PhD
Managing Editor
Abhi Dey
Consulting Editor
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Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
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